Glossary

A

A regulatory approach that aims to provide earlier access to drugs by balancing the need for timely patient access with the need for adequate evidence on the benefits and risks of a new treatment.
A type of clinical trial that allows for modifications to the trial procedures (such as dosage, sample size, or treatment duration) based on interim results, which can be particularly useful in rare diseases.
A federal agency tasked with improving the quality, safety, efficiency, and effectiveness of healthcare for all Americans.
Asking a higher court to change or reverse the decision of a lower court.
The allocation of funds for a specific purpose within government. Allows for funds to be spent but is not an actual expenditure.

B

A bill that has been introduced in both the House and Senate.
A Representative or Senator who formally signs on to support a bill. Only the first-named Member is the sponsor; all others are cosponsors, even those whose names appeared on the measure at the time it was submitted.
A measurable indicator of some biological state or condition, often used in clinical trials to identify patients who may benefit from specific treatments.
A bill that has at least one cosponsor from both parties.
A process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.

C

A body consisting of the Vice President of the United States and the heads of the executive branch’s federal executive departments.
A federal agency under the Department of Health and Human Services that works to protect public health and safety.
A federal agency that administers the nation’s major healthcare programs, including Medicare, Medicaid, and the Children’s Health Insurance Program (CHIP).
A collaborative research approach that involves community members in the research process, ensuring the research addresses community-identified needs and leads to actionable outcomes.
A bill introduced in either the House or Senate which has identical or similar language to another bill introduced in the other chamber.
Also known as Expanded Access, it refers to the use of unapproved drugs or treatments outside of clinical trials for patients with serious or immediately life-threatening conditions who have no other treatment options.
Agency within the legislative branch that produces independent analyses of budgetary and economic issues to support the Congressional process. Often calculates the cost or savings from enacting a specific bill. This is referred to as a “score.”
A type of approval in the European Union granted to a medicine that addresses unmet medical needs of patients based on less comprehensive data than normally required.
Citizens within a district of a legislator; the voters that elect a representative.

D

The Congressionally Directed Medical Research Programs that fund medical research projects.
The federal agency overseeing education and related policies in the United States.
A federal agency focused on protecting the health of all Americans and providing essential human services.
A bill that was considered and rejected by a committee; not returned to the house for action.
A database used to collect information about individuals diagnosed with specific diseases. Disease registries are vital for studying rare diseases, understanding natural history, and developing therapies.

E

When a bill is passed by both chambers and signed into law by the President.
Programs that allow patients to gain access to investigational drugs outside of clinical trials when they have a serious condition and no comparable or satisfactory alternative therapy options.

F

A process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
A federal agency responsible for protecting public health by ensuring the safety, efficacy, and security of drugs, biological products, medical devices, food, cosmetics, and radiation-emitting products.
A prolonged discussion of a piece of legislation that delays or prevents legislative action.

G

A technique that uses genes to treat or prevent disease, especially promising for many rare genetic disorders.

H

Ensuring that all people have the opportunity to attain their highest level of health, often focusing on those with unmet needs, which includes people affected by rare diseases.
A federal agency that aims to improve access to healthcare services for people who are uninsured, isolated, or medically vulnerable.
The systematic evaluation of properties, effects, and/or impacts of health technology, often focusing on the direct and intended effects of this technology, as well as its indirect and unintended consequences.
A formal meeting of a congressional committee (or subcommittee) to gather information from witnesses for use in its activities.

I

An independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services.
A network of healthcare facilities under a parent company that provides a continuum of care to a defined patient population, often integrating acute care hospitals and ambulatory services.

J

Meeting of both the House and the Senate in one chamber.

M

The political party having electoral strength sufficient to permit it to win control of a government.
Meeting by a committee or subcommittee during which committee members offer, debate, and vote on amendments to a bill or other measure.
A joint federal and state program that helps with medical costs for people with limited income and resources.
A federal health insurance program for people aged 65 and older, certain younger people with disabilities, and people with End-Stage Renal Disease.
A political party whose electoral strength is so small as to prevent its gaining control of a government except in rare and exceptional circumstances.
A proposal asking for the Senate or House to take an action.

N

A part of the U.S. Department of Health and Human Services, NIH is the nation’s medical research agency, making important discoveries that improve health and save lives.
A study that follows a group of people over time who have, or are at risk of developing, a specific medical condition to understand its progression.
Refers to organizations or entities not affiliated with or controlled by the government. Example: Patient-Centered Outcomes Research Institute (PCORI).
Not associated with a single political party or caucus.

O

A pharmaceutical agent developed specifically to treat a rare medical condition, often defined as one that affects fewer than 200,000 people in the United States.
A provision that grants exclusive marketing rights for seven years to a manufacturer upon approval of a drug for a rare disease, to encourage the development of treatments for rare diseases.

P

Being associated with a single political party or caucus.
Organizations that represent patients’ interests and provide education, support, and advocacy efforts for specific diseases or conditions.
Outcomes that matter most to patients, including quality of life, daily functioning, and other aspects that affect patients’ overall well-being.
Services provided to guide patients through the healthcare system, including managing appointments, providing education, and helping with logistics.
A type of health outcome directly reported by the patient who experienced it, without interpretation by a clinician or anyone else
A development plan aimed at ensuring that necessary data are obtained through studies in children, to support the authorization of a medicine for use in children.
A formal written request submitted by anyone other than the legislature (individuals, boards, commissions, cities, etc.).
An approach for disease treatment and prevention that considers individual variability in genes, environment, and lifestyle for each person.
A designation by the FDA that accelerates the review process for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists.
An approach for disease treatment and prevention that considers individual variability in genes, environment, and lifestyle for each person.

Q

The minimum number of members of the legislature necessary to conduct business.

R

The most senior (though not necessarily the longest-serving) member of the minority party on a committee or subcommittee. The ranking member typically oversees minority committee staff and may coordinate involvement of the minority party members in committee activities.
An international awareness day held annually on the last day of February to raise awareness for rare diseases and improve access to treatment and medical representation for individuals with rare diseases and their families.
A voucher awarded by the FDA to encourage the development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.
Clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data (RWD).
A temporary interruption in the session of Congress.
A field of medicine that aims to repair or replace damaged cells, tissues, or organs, often using stem cells or tissue engineering.
The ability of regulatory agencies to modify their requirements and processes to accommodate the unique challenges associated with developing treatments for rare diseases.
The science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of all FDA-regulated products.
A statement from a funding agency announcing the availability of grants or contracts for specific research areas.
A detailed plan submitted to regulatory agencies that outlines the activities required to ensure a medicine’s benefits outweigh its risks.

S

A pharmacy that specializes in dispensing and managing medications for patients with complex, chronic, or rare conditions.
Annual address by the President to Congress describing the current condition of the country and major policy plans, often used to highlight health initiatives.
A committee of few members of a larger committee. It is usually established for a specific purpose.
A biomarker intended to substitute for a clinical endpoint. It is used in clinical trials as a measure of how well a treatment works.

T

A specific gene, protein, or pathway that a drug or therapy aims to affect to treat a disease.
A Medicaid and Medicare program that helps people meet their healthcare needs in the community instead of going to a nursing home or other care facility.
Research that aims to translate findings in basic science into medical practice and meaningful health outcomes.

U

A disease that affects a very small number of people, often fewer than 1 in 50,000.
A statute or regulation that requires a state or local government to perform certain actions, with no money provided for fulfilling the requirements.
A condition for which there exists no satisfactory method of diagnosis, prevention, or treatment, or where existing methods are inadequate.

V

A healthcare delivery model in which providers are paid based on patient health outcomes, focusing on quality and efficiency.
The authority of the President to refuse to approve a bill or joint resolution and thus prevent its enactment into law.

W

A specialized agency of the United Nations responsible for international public health, including the development of global health policies and guidelines.

Advocacy Terms

A

B

C

D

E

F

G

H

I

J

M

N

O

P

Q

R

S

T

U

V

W